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News and press releases about the Joshua Frase Foundation and its efforts behind CNM/MTM
Fri, Sep 17, 2021
The biggest questions facing gene therapy, the XLMTM community, and Astellas after fourth patient death
After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They...
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Tue, Sep 14, 2021
Fourth boy dies in Astellas-Audentes gene therapy trial, raisingfresh fears for the field
A fourth boy has died in the Astellas-Audentes gene therapy trial for a rare muscle-wasting disease, thecompany announced Tuesday morning.
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Thu, Jul 16, 2020
A Tale of Two Clinical Trials: Gene Therapy for a Rare Disease and a Vaccine for COVID-19
Encouraging preliminary findings in a phase I clinical trial for a COVID-19 vaccine were widely reported as soon as the paper appeared in The New England Journal of...
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Tue, Jul 07, 2020
Gene Therapy: It’s Time to Talk about High-Dose AAV
A week before the 2020 virtual American Society for Gene & Cell Therapy conference, a tragedy took place in the rare disease and gene therapy communities. On May 6, the...
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Sat, Jun 27, 2020
Two patients die in now-halted study of Audentes gene therapy
Two children with a rare neuromuscular disease have died after receiving a high dose of a gene therapy in a clinical trial run by Audentes Therapeutics, according to two...
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Thu, Jun 11, 2020
Amid COVID 19 Crisis, Joshua Frase Foundation Leads Search Spanning Two Continents to Deliver Ventilator to Ailing Child in Ecuador
One-Year-Old With Rare Centronuclear Myopathy Diagnosis Gets Life Saving Ventilator Thanks To Dauntless Network of Families in U.S. and Europe.
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Wed, Sep 18, 2019
The importance of patient registries. Recently, the Joshua Frase Foundation's patient registry was used in collaboration in this published paper, RECENSUS.
Mortality and respiratory support in X-linked myotubular myopathy: a RECENSUS retrospective analysis
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Tue, May 07, 2019
How this promising gene therapy for a rare neuromuscular disease was fueled by passionate parents and a dog
Many treatments for rare diseases begin with families who work tirelessly, sometimes for decades, to fund the initial studies leading to the clinic. For X-linked
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Wed, May 01, 2019
Audentes Therapeutics Presents New Positive Data from ASPIRO, the Phase 1/2 Clinical Trial of AT132 for X-linked Myotubular Myopathy, at 22nd Annual Meeting of the American Society of Gene and Cell Therapy
New data include 48 weeks of follow-up for six treated patients in dose Cohort 1 and 24 weeks for three treated patients in dose Cohort 2 ...
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Tue, Apr 30, 2019
Dynacure Announces Approval of Clinical Trial Application for DYN101, an Antisense Medicine to Treat Rare Disease ‘Centronuclear Myopathies’
Company expects to initiate Phase 1 / 2 study 'Unite-CNM' in 2H2019
Strasbourg (France), April 30, 2019
Dynacure, a...
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Mon, Jul 09, 2018
The Matriarch of the First MTM Dog Colony...The Power of Patient Advocacy
The Joshua Frase Foundation (JFF) is a non-profit organization founded with a simple yet seemingly unattainable vision: to find a cure for Myotubular Myopathy (MTM). The...
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Thu, Jun 21, 2018
Rare Leader: Alison Rockett Frase, President of the Joshua Frase Foundation
The Basics Name : Alison Rockett Frase Title : President, co-founder, and patient advocate ...
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Thu, Jun 07, 2018
Gene Therapy for Myotubular Myopathy: Early Signs of Success!
Parents cherish developmental milestones, from a newborn’s grip of an offered finger; to an infant’s holding her head up the first time; to rolling over, creeping, and...
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Wed, Feb 28, 2018
A Boy With the Will to Live: Biobanking Contributing to Breakthroughs in Rare Diseases
Joshua Frase was born on February 2, 1995. When Joshua was three months old, a muscle biopsy revealed that he had the ultra-rare X-linked myotubular myopathy (XLMTM), a...
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Wed, Nov 22, 2017
UW, Family Rewriting Neuromuscular Research
Physicians and researchers at the UW Medicine Institute for Stem Cell and Regenerative Medicine have partnered up with a nonprofit foundation in one of the leading efforts to...
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Tue, Oct 17, 2017
Transnetyx produces "Brick in the Wall," second short film in its Love Letter Series
MEMPHIS, Tenn., Oct. 17, 2017 /PRNewswire/ -- Transnetyx, an automated genotyping company that serves biomedical researchers worldwide, today announced the premier of the...
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Wed, Oct 11, 2017
The Joshua Frase Foundation for Congenital Myopathy Research, Inc.
Article on Alison's speech at the National Animal Interest Alliance, October 2017
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Thu, May 18, 2017
KIRO 7 Investigates Revolutionary Treatment in Dogs to be Tested in Children
Our recent campaign to increase participation in our Family Registry was published in MDA Quest Magazine, and can be seen below.
